Personalized Medicine: Changing the Paradigm for Value

San Diego—Personalized medicine is promising to revolutionize medical care by tailoring diagnostic and therapeutic approaches to individual patients based on an improved understanding of genetics and molecular biology. For pharmacists and pharmacy groups, this focus on personalized medicine will pose a number of challenges to delivering care. Among these will be issues regarding the economics of personalized medicine, including coding and reimbursement issues, as well as formulary management for high cost, targeted therapies.

These issues were discussed during a Contemporary Issues session at the AMCP meeting. The session was titled How Personalized Medicine Is Changing the Paradigm for Establishing Value Using Real-World Data.

Requirement for Changes to Evidence as Personalized Medicine Expands

In the opening session, Diana I. Brixner, RPh, PhD, professor and chair, department of pharmacotherapy, University of Utah, Salt Lake City, provided 2 examples that show how genetic information is being used to tailor treatment in different clinical settings. Access to genetic information and advanced therapies in oncology has already led to targeted therapies at increasing cost, which, in turn, she said, has led to a greater focus on evidence-based guidelines and treatment pathways for cancer management. A next step is to assess treatment effectiveness based on this evidence in terms of real-world outcomes.

In a second example, she cited a Danish prospective cohort study [Clinical Epidemiology 2012;4(suppl 1):7-13] underway to develop genetic risk profiles of patients with type 2 diabetes, and to assess the impact of these profiles on treatment and outcomes due to disease etiology, drug kinetics, and metabolism.

Dr. Brixner also provided an update on the integration of higher standards for molecular diagnostics for evaluation by the FDA. To date, she said, the FDA has reviewed a lot of therapeutic development programs with potential companion diagnostics (Dx) and more than 15 companion Dx applications. None of these programs or products are alike, she said, adding that codevelopment guidance is needed to describe points to consider in both therapeutic and diagnostic development programs as well as describe FDA preferences for certain elements. She said that one particular development pathway would not be prescribed.

Comparative Effectiveness Evidence and Issues for Reimbursement

Susan Garfield, DrPH, vice president of market access and health care, GfK Bridgehead, Wayland, Massachusetts, spoke on the complexity of optimizing reimbursement in the United States and said that positive outcomes increasingly depend on quality of clinical and economic evidence. She emphasized that evidence requirements impact every level of market access and coverage for personalized medicine products. For example, regulatory requirements are focusing on evolving pathways for companion products and parallel reviews between the FDA and the Centers for Medicare & Medicaid Services are beginning to be seen. Changes in payment are also emerging with increased patient cost-sharing and bundled payments. Insurance coverage is also increasingly requiring comparative effectiveness data and clinical utility benchmarks.

As comparative effectiveness data take on an increasingly important role in personalized medicine, payers are trying to translate the information gained from this evidence into insights, Dr. Garfield said. For example, payers are working with industry to track impact using claims data and real-time reporting as well as leveraging big data for insights beyond short-term clinical outcomes. Overall, payers still want to know what is the most valuable use of limited healthcare resources.

Going forward, these evidence requirements for personalized medicine will likely set the standard for industry. As such, Dr. Garfield posed a number of questions to consider. “Personalized medicine will clarify treatment pathways for some patients, while leaving others without clear pathways,” she said. “Do manufacturers have to develop evidence that follows not indicated patient groups for true comparison?” She also questioned how regulators should address real-world versus trial data, and whether big data should impact conceptions of safety and efficacy.

Formulary Management and Personalized Medicine

H. Eric Cannon, PharmD, FAMCP, chief of pharmacy and director of health and wellness for SelectHealth, Salt Lake City, Utah, provided examples of formulary management that incorporate real-world data on personalized medicine. He cited a study that looked at the appropriateness of criteria developed by the American Academy of Pediatrics to determine prior authorization (PA) of palivizumab for treatment of respiratory syncytial virus (RSV) in children [J Manag Care Pharm 2010;16(1):15-22].

The study assessed hospitalization rates associated with RSV infection, incidence of emergency department visits, deaths, and costs. The study found that the PA program resulted in a drug cost avoidance of $2.4 million in a 500,000 member health plan, and that the savings on drug costs were achieved without apparent adverse clinical outcomes in the rate of emergency room visits or rate of RSV-related hospitalizations.

This example and others, Dr. Cannon said, show that real-world evidence, which is specific to individual patients and risk factors, can be applied. He urged health plans to develop a knowledge base to collect and use real-world evidence in the application of personalized medicine, and emphasized the need for formulary decisions to be fluid and for plans to have the ability to change programs as evidence emerges.